An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study

This study is designed to provide continued access to BCX9930 for participants currently receiving treatment with BCX9930 in a BioCryst-sponsored clinical study for paroxysmal nocturnal hemoglobinuria (PNH) who, in the opinion of the investigator, would benefit from continued treatment with BCX9930 and who do not have access to other treatment options.

Start Date18 Jan 2023

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

NCT05162066 / TerminatedPhase 2

An Open-Label, Safety, Tolerability, and Proof-of-Concept Study of Oral BCX9930 Therapy in Subjects With Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy, or Primary Membranous Nephropathy

The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.

Start Date18 Feb 2022

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

NCT05116774 / TerminatedPhase 2

A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.

Start Date06 Dec 2021

Sponsor / Collaborator

BioCryst Pharmaceuticals, Inc.

100 Clinical Results associated with Pelecopan

100 Translational Medicine associated with Pelecopan

100 Patents (Medical) associated with Pelecopan

Literatures (Medical) associated with Pelecopan

19 Jul 2024·MEDICINE

A review of progress on complement and primary membranous nephropathy

Review

Author: Sun, Jia ; Yu, Shanshen

Primary membranous nephropathy (PMN) is a predominant cause of adult nephrotic syndrome, with its incidence witnessing a progressive surge over time. Approximately 35% to 47% of patients progress to renal failure within 10 years, causing a huge social burden. Within China, the proportion of PMN in primary glomerular disease exhibits a gradual ascension. Recent studies have shown that the 3 activation pathways of complement: the classical pathway, mannose-binding lectin pathway, and alternative pathway, are all involved in the pathogenesis of PMN. Despite historical limitations in detecting C1q deposits on the glomeruli of PMN in the past, recent studies have confirmed the classical pathway is implicated in patients with PMN. Considering the dysregulation of the complement system has been observed in PMN, complement inhibitors become increasingly promising. Several clinical trials are presently underway to evaluate the efficacy of complement inhibitors, such as MASP2 antagonists (OMS721), C3 and C3b antagonists (APL2), FD inhibitors (BCX9930), C3aR antagonists (SB290157 and JR14a), FB inhibitors (LNP023). This article reviews the recent research progress on the role of the complement pathway in the pathogenesis of PMN, and underscores the importance of continued research into the complement pathway and its inhibitors, which may pave the way for groundbreaking advancements in the management of PMN.

Pharmaceuticals (Basel, Switzerland)

Complement System as a New Target for Hematopoietic Stem Cell Transplantation-Related Thrombotic Microangiopathy

Review

Author: Ardissino, Gianluigi ; Porcaro, Luigi ; Capone, Valentina ; Tedeschi, Silvana ; Cugno, Massimo

Thrombotic microangiopathy (TMA) is a complication that may occur after autologous or allogeneic hematopoietic stem cell transplantation (HSCT) and is conventionally called transplant-associated thrombotic microangiopathy (TA-TMA). Despite the many efforts made to understand the mechanisms of TA-TMA, its pathogenesis is largely unknown, its diagnosis is challenging and the case-fatality rate remains high. The hallmarks of TA-TMA, as for any TMA, are platelet consumption, hemolysis, and organ dysfunction, particularly the kidney, leading also to hypertension. However, coexisting complications, such as infections and/or immune-mediated injury and/or drug toxicity, together with the heterogeneity of diagnostic criteria, render the diagnosis difficult. During the last 10 years, evidence has been provided on the involvement of the complement system in the pathophysiology of TA-TMA, supported by functional, genetic, and therapeutic data. Complement dysregulation is believed to collaborate with other proinflammatory and procoagulant factors to cause endothelial injury and consequent microvascular thrombosis and tissue damage. However, data on complement activation in TA-TMA are not sufficient to support a systematic use of complement inhibition therapy in all patients. Thus, it seems reasonable to propose complement inhibition therapy only to those patients exhibiting a clear complement activation according to the available biomarkers. Several agents are now available to inhibit complement activity: two drugs have been successfully used in TA-TMA, particularly in pediatric cases (eculizumab and narsoplimab) and others are at different stages of development (ravulizumab, coversin, pegcetacoplan, crovalimab, avacopan, iptacopan, danicopan, BCX9930, and AMY-101).

News (Medical) associated with Pelecopan

05 Aug 2024

·www.globenewswire.com

BioCryst Reports Second Quarter 2024 Financial Results and Provides Business Update

—Q2 2024 ORLADEYO net revenue grows 34 percent y-o-y to $108.3 million— —Full-year 2024 ORLADEYO revenue guidance increased to $420-$435 million (previously $390-$400 million)— —Company generates GAAP operating profit of $8.8 million in second quarter ($21.9 million non-GAAP operating profit)— —Pipeline continues to advance— RESEARCH TRIANGLE PARK, N.C., Aug. 05, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the second quarter ended June 30, 2024, and provided a corporate update. “The first half of 2024 has been outstanding for BioCryst due to the success we are having in the marketplace with ORLADEYO. As a result, we are increasing our full year guidance for ORLADEYO, advancing multiple programs toward the clinic in the next 18 months and moving the company closer to profitability,” said Jon Stonehouse, president and chief executive officer of BioCryst. ORLADEYO® (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks ORLADEYO net revenue in the second quarter of 2024 was $108.3 million (+34 percent year-over-year (y-o-y)).Operational improvements drove revenue above expectations for the second quarter. This included a three percent increase in the overall rate of paid patients relative to the end of 2023, which now stands at 74.4 percent.New patient starts continued at the same high rate seen in the prior two quarters, matching the new patient demand seen in the first three quarters of the launch in 2021, and patient retention remained strong. We continue to see more than 60 percent of patients on paid therapy staying on for at least a year.A recent market research study showed that 52 percent of allergist/immunologists are extremely likely to prescribe ORLADEYO to more patients, up from 29 percent in early 2023.With additional recent approvals and reimbursement authorizations in Europe and Latin America, ORLADEYO is now commercially available to patients in more than 20 countries. Ex-U.S. ORLADEYO revenue in the second quarter increased 51 percent y-o-y and accounted for 11 percent of global ORLADEYO net revenues. “Our second quarter revenue growth and increased full year guidance for ORLADEYO reflect strong underlying demand and favorable patient outcomes, combined with our continuing focus on improving patient services and market access operations. The real-world experience of patients who respond to ORLADEYO is consistent with the 91 percent reduction in attacks from baseline that we saw in long-term clinical trials. The potential for patients to have that high level of HAE attack control in a once-daily pill is a major differentiator in the marketplace that makes ORLADEYO’s trajectory toward $1 billion in peak sales increasingly clear,” said Charlie Gayer, chief commercial officer of BioCryst. Rare Disease Pipeline The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients. The company remains on track to submit a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive an oral granule formulation of ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE.The company has completed its clinical evaluation of its oral Factor D inhibitor, BCX10013. The drug was safe and well tolerated at all doses studied, however the level of clinical activity observed was less than other therapies on the market and potential partners have declined to make the additional investment required to evaluate higher doses. BioCryst plans to discontinue development, consistent with its previously announced plans.The company expects to advance BCX17725, its KLK-5 inhibitor for the treatment of Netherton syndrome, into the clinic by the end of 2024. Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair and immune system. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there is no approved treatment for Netherton syndrome. In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME). DME is an important cause of vision loss in diabetes and is due to leakage from the blood vessels in the retina. While current treatments focus on VEGF inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space as a depot formulation, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system. Second Quarter 2024 Financial Results For the three months ended June 30, 2024, total revenues were $109.3 million, compared to $82.5 million in the second quarter of 2023 (+32.5 percent year-over-year (y-o-y)). The increase was primarily due to $108.3 million in ORLADEYO net revenue in the second quarter of 2024, compared to $81.0 million in ORLADEYO net revenue in the second quarter of 2023 (+33.7 percent y-o-y). R&D expenses for the second quarter of 2024 decreased to $37.6 million from $51.2 million in the second quarter of 2023 (-26.6 percent y-o-y), primarily due to decreased spending on BCX10013 and the discontinuation of the BCX9930 program. These reductions were partially offset by increased investment in BCX17725, avoralstat and other discovery programs, and our ongoing ORLADEYO pediatric trial. Selling, general and administrative expenses for the second quarter of 2024 increased to $61.2 million, compared to $51.0 million in the second quarter of 2023 (+20.0 percent y-o-y), primarily due to an increase in commercial expenses to support growing revenue, newly launched regions and expanded international operations. In addition, there was an increase in general and administrative expenses, primarily related to an increase in resourcing to support our accounting and IT functions. Total operating expenses were $100.6 million for the second quarter of 2024, compared to $103.2 million in the second quarter of 2023 (-2.5 percent y-o-y). Non-cash stock compensation was $13.2 million for the second quarter of 2024. Total operating expenses, not including non-cash stock compensation for the second quarter of 2024 were $87.4 million, compared to $90.4 million in the second quarter of 2023 (-3.3 percent y-o-y). The company generated a GAAP operating profit of $8.8 million in the second quarter of 2024. This compares to a GAAP operating loss of $20.7 million in the second quarter of 2023. Adjusted for non-cash stock compensation, the non-GAAP operating profit was $21.9 million in the second quarter of 2024, compared to a non-GAAP operating loss of $7.9 million in the second quarter of 2023. Interest expense was $24.7 million in the second quarter of 2024, compared to $28.9 million in the second quarter of 2023 (-14.5 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations. Net loss for the second quarter of 2024 was $12.7 million, or $0.06 per share, compared to a net loss of $75.3 million, or $0.40 per share, for the second quarter of 2023. In the second quarter of 2023, there was a $29.0 million one-time debt extinguishment fee related to the close-out of the Athyrium debt facility. Excluding this one-time event, non-GAAP net loss for the second quarter of 2023 was $0.24 per share. Cash, cash equivalents, restricted cash and investments totaled $338.1 million at June 30, 2024, compared to $415.7 million at June 30, 2023. Operating cash use for the second quarter of 2024 was $0.2 million. Non-GAAP Pro forma Financial Measures The information furnished in this release includes non-GAAP pro forma financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (GAAP), including financial measures labeled as “non-GAAP” or “adjusted.” We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allow investors to more accurately understand our second quarter 2023 and second quarter 2024 results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expected Wall Street analysts to compare our results. Our non-GAAP pro forma measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue, operating income, net income, operating profit and earnings per share. Our references to our second quarter 2023 and first six months 2023 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time loss on the extinguishment of the Athyrium term loans. Our reference to our second quarter 2024 and first six months 2024 “non-GAAP pro forma” financial measure of non-GAAP operating profit constitutes a non-GAAP financial measure. It refers to our GAAP results, adjusted to show the results without including non-cash stock compensation expense. Financial Outlook for 2024 Based on the operational improvements and strong patient and physician demand for ORLADEYO seen in the first half of 2024, the company is raising its outlook for full year 2024 global net ORLADEYO revenue to be between $420 million and $435 million (previously $390 million to $400 million). The company maintains its prior operating expense outlook, and expects full year 2024 operating expenses to be between $365 million and $375 million, flat to full year 2023 operating expenses. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024. Based on the company’s disciplined approach to capital allocation, and the increased revenue guidance for ORLADEYO, the company is confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation) and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon. Conference Call and WebcastBioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; risks related to the planned discontinuation of the development of BCX10013; BioCryst’s ability to successfully progress its pipeline development plans as described herein; risks related to the reduction in size of BioCryst’s R&D organization; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com BIOCRYST PHARMACEUTICALS, INC.CONSOLIDATED FINANCIAL SUMMARY(In thousands, except per share) Statements of Operations (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenues: ORLADEYO $108,288 $81,009 $197,155 $149,423 Other 1,044 1,482 4,938 1,846 Total revenues 109,332 82,491 202,093 151,269 Expenses: Cost of product sales 1,699 894 2,964 1,825 Research and development 37,623 51,247 84,116 99,635 Selling, general and administrative 61,214 50,997 120,578 98,864 Royalty 35 56 162 63 Total operating expenses 100,571 103,194 207,820 200,387 Income (loss) from operations 8,761 (20,703) (5,727) (49,118) Interest income 3,554 3,750 7,585 7,128 Interest expense (24,733) (28,915) (49,239) (56,311)Foreign currency (losses) gains, net (84) 301 (135) 72 Loss on extinguishment of debt — (29,019) — (29,019)Loss before income taxes $(12,502) $(74,586) $(47,516) $(127,248)Income tax expense 172 740 537 1,411 Net loss $(12,674) $(75,326) $(48,053) $(128,659) Basic and diluted net loss per common share $(0.06) $(0.40) $(0.23) $(0.68) Weighted average shares outstanding 206,425 189,118 206,244 188,815 Balance Sheet Data (in thousands) June 30, 2024 December 31, 2023 (unaudited) (Note 1)Cash, cash equivalents and investments $336,344 $388,987 Restricted cash 1,795 1,804 Receivables 68,759 56,950 Total assets 472,419 516,960 Secured term loan 313,822 303,231 Royalty financing obligation 523,633 531,599 Accumulated deficit (1,729,212) (1,681,159)Stockholders’ deficit (475,606) (455,528)Shares of common stock outstanding 206,629 205,771 Note 1: Derived from audited financial statements. Reconciliation of Adjusted Net Loss and Adjusted Diluted Earnings Per Share (in thousands) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 GAAP net loss $(12,674) $(75,326) $(48,053) $(128,659)Less: One-time R&D restructuring expense — — (1,264) — Less: One-time loss on extinguishment of Athyrium term loans — (29,019) — (29,019)Adjusted net loss $(12,674) $(46,307) $(46,789) $(99,640) GAAP basic and diluted net loss per common share $(0.06) $(0.40) $(0.23) $(0.68) Adjusted basic and diluted net loss per common share $(0.06) $(0.24) $(0.23) $(0.53) Reconciliation of Adjusted Income (Loss) from Operations (in thousands) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 GAAP income (loss) from operations $8,761 $(20,703) $(5,727) $(49,118)Less: Stock-based compensation expense (13,173) (12,841) (26,825) (26,848)Adjusted income (loss) from operations $21,934 $(7,862) $21,098 $(22,270)

Financial StatementDrug Approval

06 May 2024

·www.biospace.com

BioCryst Reports First Quarter 2024 Financial Results and Provides Business Update

Q1 2024 ORLADEYO net revenue grows 30 percent y-o-y to $88.9 million Full-year 2024 ORLADEYO revenue guidance adjusted to $390-$400 million (top end of prior guidance range) Pipeline programs, including BCX17725 for Netherton syndrome and avoralstat for DME, advancing on schedule into clinical trials RESEARCH TRIANGLE PARK, N.C., May 06, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the first quarter ended March 31, 2024, and provided a corporate update. “We are off to a fantastic start to the year with outstanding ORLADEYO revenue growth and our prioritized pipeline programs advancing on schedule. We are focused on continuing this momentum as we see strong patient demand for ORLADEYO and more pipeline programs advancing into the clinic, starting later this year,” said Jon Stonehouse, president and chief executive officer of BioCryst. ORLADEYO® (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks ORLADEYO net revenue in the first quarter of 2024 was $88.9 million (+30 percent year-over-year (y-o-y)). In the first quarter, the U.S. commercial team accelerated patients going through annual reimbursement reauthorization from free drug to paid drug faster than in previous years, generating higher than expected ORLADEYO revenue. New patient growth remained strong, with the past two quarters having the most new prescriptions in the United States since the first two quarters of the launch. Sales from outside the U.S. contributed 10 percent of global ORLADEYO net revenues in the first quarter, as the number of patients treated with ORLADEYO continued to grow strongly and consistently in new and existing markets. New real-world evidence showing significant reductions in healthcare resource utilization among patients with HAE following ORLADEYO initiation to be presented in a poster on May 8 at the 2024 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR). “Our team made exceptional efficiency improvements in the U.S. prescription reauthorization process, and new prescriptions remained very strong as physicians and patients increasingly understand that ORLADEYO provides excellent efficacy and convenience. These two successes helped us exceed our revenue projections for the quarter and, as a result, we are increasing our revenue guidance for 2024,” said Charlie Gayer, chief commercial officer of BioCryst. Rare Disease Pipeline The goal with our pipeline is to continue bringing selected, highly differentiated rare disease products to the market, and to reproduce the commercial success we have delivered with ORLADEYO. Milestones in the next 18 months include: The ongoing proof-of-concept trial with BCX10013, an oral Factor D inhibitor, remains on track. The company expects to partner or discontinue the program later this year. Enrollment is complete in the APeX-P pediatric trial. Data from the trial will support a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE. The company expects to advance BCX17725, its KLK-5 inhibitor for the treatment of Netherton syndrome, into the clinic by the end of 2024. Netherton syndrome is a rare, lifelong genetic disorder that often presents in neonates or infancy with red, scaly and inflamed skin and susceptibility to recurrent skin infections. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there is no approved treatment for Netherton syndrome. In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME). DME is the most common cause of vision loss in individuals with diabetes and at least one-third of patients have persistent DME despite anti-VEGF therapies, which are administered via monthly injection. By delivering avoralstat with Clearside’s SCS microinjector® directly into the suprachoroidal space of the eye in the clinical trial, avoralstat could inhibit plasma kallikrein at the sites of edema formation in DME disease, the retinal and choroidal vascular endothelium. With its low solubility, the drug could persist in the eye at the site of disease for a long duration, resulting in less frequent injections. “It is very exciting to be advancing our pipeline of first-in-class/best-in-class molecules into the clinic to generate proof-of-concept data across multiple programs and diseases, and to be so close to delivering the first oral prophylactic option to children with HAE,” said Dr. Helen Thackray, chief research and development officer of BioCryst. First Quarter 2024 Financial Results For the three months ended March 31, 2024, total revenues were $92.8 million, compared to $68.8 million in the first quarter of 2023 (+34.9 percent y-o-y). The increase was primarily due to $88.9 million in ORLADEYO net revenue in the first quarter of 2024, compared to $68.4 million in ORLADEYO net revenue in the first quarter of 2023 (+30 percent y-o-y). Research and development (R&D) expenses for the first quarter of 2024 decreased to $46.5 million from $48.4 million in the first quarter of 2023 (-3.9 percent y-o-y), primarily due to decreased spending on BCX10013 due to our plan to either out-license or discontinue late-stage development and commercialization, which was announced in January 2024, as well as the discontinuation of the BCX9930 program, which was announced in December 2022. These reductions were partially offset by increased investment in BCX17725 and other discovery programs, and ORLADEYO label expansion and life cycle investments, such as our ongoing ORLADEYO pediatric trial. Selling, general and administrative (SG&A) expenses for the first quarter of 2024 increased to $59.4 million, compared to $47.9 million in the first quarter of 2023 (+24.0 percent y-o-y). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and expand and support international operations. Operating loss for the first quarter was $14.5 million, adjusted to $0.8 million excluding non-cash stock compensation. Interest expense was $24.5 million in the first quarter of 2024, compared to $27.4 million in the first quarter of 2023 (-10.6 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations, partially offset by an increase in interest expense associated with the Pharmakon debt refinancing secured in April 2023. Net loss for the first quarter of 2024 was $35.4 million, or $0.17 per share, compared to a net loss of $53.3 million, or $0.28 per share, for the first quarter of 2023. Non-GAAP net loss for the first quarter of 2024 was $34.1 million, or $0.17 per share when excluding one-time costs associated with the R&D restructuring, totaling $1.3 million. A reconciliation between GAAP and non-GAAP net loss is provided in the table below. Cash, cash equivalents, restricted cash and investments totaled $338.4 million at March 31, 2024, compared to $403.1 million at March 31, 2023. Net cash utilization for the first quarter of 2024 was $52.4 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense. We expect, as in previous years, that the first quarter will be the highest quarter of operating cash use for the year, and that operating cash use for the remaining quarters of 2024 will normalize around $10-$12 million per quarter. We expect that total cash at the end of 2024 will be above $300 million. Non-GAAP Pro forma Financial Measures The information furnished in this release includes non-GAAP pro forma financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (“GAAP”), including financial measures labeled as “non-GAAP” or “adjusted.” We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allowed investors to more accurately understand our current period results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP pro forma measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue, operating income, net income, and earnings per share. Our references to our first quarter 2024 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time costs associated with the R&D restructuring. A reconciliation between GAAP and non-GAAP net loss is provided in the table below. Financial Outlook for 2024 Based on the team’s success in accelerating ORLADEYO patients through the U.S. reauthorization process faster than anticipated, the company is adjusting its outlook for full year 2024 global net ORLADEYO revenue to be between $390 million and $400 million, the top end of its prior guidance range. The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to full year 2023 operating expenses. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024. Based on the company’s disciplined approach to capital allocation, and the adjusted revenue guidance for ORLADEYO, the company is even more confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon. Conference Call and Webcast BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at . About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; BioCryst’s ability to successfully implement its plans for BCX10013, including that the results of the ongoing proof-of-concept trial may differ from BioCryst’s expectations and that BioCryst may not be able to successfully out-license the late-stage development and commercialization of BCX10013; BioCryst’s ability to successfully progress its pipeline development plans as described herein; risks related to the reduction in size of BioCryst’s R&D organization; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact: John Bluth +1 919 859 7910 jbluth@biocryst.com BIOCRYST PHARMACEUTICALS, INC. CONSOLIDATED FINANCIAL SUMMARY (In thousands, except per share) Statements of Operations (unaudited) Three Months Ended March 31, 2024 2023 Revenues: ORLADEYO $ 88,867 $ 68,414 Other 3,894 364 Total revenues 92,761 68,778 Expenses: Cost of product sales 1,265 931 Research and development 46,493 48,388 Selling, general and administrative 59,364 47,867 Royalty 127 7 Total operating expenses 107,249 97,193 Loss from operations (14,488 ) (28,415 ) Interest income 4,031 3,378 Interest expense (24,506 ) (27,396 ) Foreign currency losses, net (51 ) (229 ) Loss before income taxes $ (35,014 ) $ (52,662 ) Income tax expense 365 671 Net loss $ (35,379 ) $ (53,333 ) Basic and diluted net loss per common share $ (0.17 ) $ (0.28 ) Weighted average shares outstanding 206,064 188,509 Balance Sheet Data (in thousands) March 31, 2023 (unaudited) December 31, 2023 (Note 1) Cash, cash equivalents and investments $ 336,554 $ 388,987 Restricted cash 1,798 1,804 Receivables 60,586 56,950 Total assets 467,892 516,960 Secured term loan 308,484 303,231 Royalty financing obligation 530,574 531,599 Accumulated deficit (1,716,538 ) (1,681,159 ) Stockholders’ deficit (476,167 ) (455,528 ) Shares of common stock outstanding 206,347 205,771 Note 1: Derived from audited financial statements. Reconciliation of Adjusted Net Loss and Adjusted Diluted Earnings Per Share (in thousands) Three Months Ended March 31, 2024 2023 GAAP net loss $ (35,379 ) $ (53,333 ) Less: One-time R&D restructuring expense (1,264 ) — Adjusted net loss $ (34,115 ) $ (53,333 ) GAAP basic and diluted net loss per common share $ (0.17 ) $ (0.28 ) Adjusted basic and diluted net loss per common share $ (0.17 ) $ (0.28 )

Financial Statement

26 Feb 2024

·www.globenewswire.com

BioCryst Reports Fourth Quarter and Full Year 2023 Financial Results and Upcoming Key Milestones

—ORLADEYO net revenue of $90.9 million for Q4 2023 and $326.0 million for FY 2023 (+30 percent y-o-y) — —2024 ORLADEYO net revenue expected to be between $380-$400 million— —Company expects operating profit in 2024, approaching positive EPS and positive cash flow in 2H 2025— RESEARCH TRIANGLE PARK, N.C., Feb. 26, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update. “The impressive growth we are seeing with ORLADEYO has put us in a position to accelerate our path to profitability while continuing to invest in our diverse pipeline of first-in-class or best-in-class molecules that we believe will deliver our next marketed product,” said Jon Stonehouse, president and chief executive officer of BioCryst. Program Updates and Key Milestones ORLADEYO® (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks ORLADEYO net revenue in the fourth quarter of 2023 was $90.9 million. The total number of U.S. patients on paid or long-term free product reached 1,104 at the end of the fourth quarter (+30 percent y-o-y) with 71.5 percent of those patients on paid product.Net U.S. patient growth totaled 321 in 2023, including new patients still on short-term quick start product at the end of the quarter.The number of new ORLADEYO prescribers in the fourth quarter of 2023 (Q4 2023) was the largest number of new prescribers of any quarter in 2023. Real-world data presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting reinforced prior data showing patients switching to ORLADEYO experience sustained attack reduction regardless of baseline attack rate or prior HAE prophylaxis treatment.Access to ORLADEYO continues to expand to more HAE patients around the world. In Q4 2023, ORLADEYO was approved in Argentina, launched in Spain, and granted final pricing approval in Austria. In the first quarter of 2024, ORLADEYO secured final reimbursement in Italy. “As the real-world evidence with ORLADEYO consistently underscores, patients are switching to ORLADEYO because they can achieve outstanding HAE attack control and tolerability with an oral, once-daily therapy, leading to life-changing results,” said Charlie Gayer, chief commercial officer of BioCryst. Fourth Quarter 2023 Financial Results For the three months ended December 31, 2023, total revenues were $93.4 million, compared to $79.5 million in the fourth quarter of 2022. The increase was primarily due to $90.9 million in ORLADEYO net revenue in the fourth quarter of 2023, compared to $70.7 million in the fourth quarter of 2022. Revenue in the fourth quarter of 2023 also included $2.3 million of net revenue from RAPIVAB related sales, compared to $8.7 million in the fourth quarter of 2022. Research and development (R&D) expenses for the fourth quarter of 2023 decreased to $70.1 million from $73.2 million in the fourth quarter of 2022 (-4 percent year-over-year), primarily due to the discontinuation of the BCX9930 and BCX9250 programs announced in December 2022 and November 2022, respectively. These reductions were partially offset by increased investment in BCX17725 and our other early-stage programs, including the $5 million upfront payment to Clearside Biomedical related to avoralstat, and ORLADEYO label expansion and life cycle investments, such as our ongoing ORLADEYO pediatric trial. Selling, general and administrative (SG&A) expenses for the fourth quarter of 2023 increased to $64.4 million, compared to $50.2 million in the fourth quarter of 2022 (+28 percent year-over-year). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and international operations. Interest expense was $24.6 million in the fourth quarter of 2023, compared to $26.5 million in the fourth quarter of 2022. The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations, partially offset by an increase in interest expense associated with the Pharmakon debt refinancing secured in April 2023. Net loss for the fourth quarter of 2023 was $61.7 million, or $0.31 per share, compared to a net loss of $71.5 million, or $0.38 per share, for the fourth quarter of 2022. Non-GAAP net loss for the fourth quarter of 2023 was $56.4 million, or $0.28 per share when excluding one-time costs associated with the R&D restructuring and the postponement of the expansion at our Discovery Center in Alabama, totaling $5.4 million. A reconciliation between GAAP and non-GAAP net loss is provided in the table below. Cash, cash equivalents, restricted cash and investments totaled $390.8 million as of December 31, 2023, compared to $443.9 million as of December 31, 2022. Net cash utilization for the fourth quarter of 2023 was $8.6 million. Full Year 2023 Financial Results For the full year ended December 31, 2023, total revenues were $331.4 million, compared to $270.8 million in the full year ended December 31, 2022. The increase was primarily due to $326.0 million of ORLADEYO net revenue in 2023, compared to $251.6 million in 2022. The increase in ORLADEYO net revenue was partially offset by a decrease in other net revenues of $13.8 million, primarily due to RAPIVAB stockpiling sales to complete the U.S. Department of Health and Human Services procurement contract during the year ended December 31, 2022. R&D expenses in full year 2023 decreased to $216.6 million from $253.3 million in full year 2022 (-14 percent year-over-year), primarily due to the discontinuation of the BCX9930 and BCX9250 programs announced in December 2022 and November 2022, respectively. These reductions were partially offset by increased investment in BCX17725, increased investment in our other early-stage discovery programs, including the $5 million upfront payment to Clearside Biomedical related to avoralstat, ORLADEYO label expansion and life cycle investments, such as our ongoing ORLADEYO pediatric trial, and an increase in indirect costs to support our programs. SG&A expenses in full year 2023 increased to $213.9 million, compared to $159.4 million in full year 2022 (+34 percent year-over-year). The increase was primarily due to increased investment in order to expand and enhance the U.S. commercial team and expanded international operations. Interest expense was $108.2 million in full year 2023, compared to $99.1 million in full year 2022. The increase in interest expense was primarily associated with the interest accrued on the Tranche A Loan of $300.0 million under the Pharmakon Loan Agreement. Other expense was comprised primarily of a loss on extinguishment of debt of $29.0 million on the repayment of the term loans under the Athyrium Credit Agreement and net foreign currency losses of $1.0 million, partially offset by interest income of $15.8 million for the year ended December 31, 2023. Other income was comprised of interest income of $5.1 million, partially offset by net foreign currency losses of $2.0 million for the year ended December 31, 2022. Net loss for full year 2023 was $226.5 million, or $1.18 per share, compared to a net loss of $247.1 million, or $1.33 per share, for full year 2022. Non-GAAP net loss for full year 2023 was $192.2 million, or $1.00 per share when excluding the one-time loss on debt extinguishment of $29.0 million on the repayment of the term loans under the Athyrium Credit Agreement recognized in the second quarter of 2023, as well as the R&D restructuring and the postponement of previously planned capital expenditures at our Discovery Center in Alabama recognized in the fourth quarter of 2023, totaling $5.4 million. A reconciliation between GAAP and non-GAAP net loss is provided in the table below. Non-GAAP Pro forma Financial Measures The information furnished in this release includes non-GAAP pro forma financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (“GAAP”), including financial measures labeled as “non-GAAP” or “adjusted.” We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allowed investors to more accurately understand our 2023 results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP pro forma measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue, operating income, net income, and earnings per share. Our references to our fourth quarter 2023 and full year 2023 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time loss on debt extinguishment on the repayment of the term loans under the Athyrium Credit Agreement, as well as the R&D restructuring and the postponement of previously planned capital expenditures at our Discovery Center in Alabama. A reconciliation between GAAP and non-GAAP net loss is provided in the table below. Financial Outlook for 2024 The company expects full year 2024 global net ORLADEYO revenue to be between $380 million and $400 million. The general pattern of revenue throughout 2024 is expected to be similar to past years, with the seasonal impact of prescription reauthorizations and the potential impact of the Inflation Reduction Act in the first quarter driving a quarter-over-quarter revenue decline in the first quarter, followed by a strong return to growth in the second quarter. The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to expected full year 2023 operating expenses. The company now expects that R&D expenses in 2024 will be reduced by between $20 million and $30 million versus 2023. SG&A expenses are expected to increase by $20 million in 2024, primarily to support the continued U.S. and global growth of ORLADEYO to $1 billion in peak sales. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024. Based on the company’s disciplined approach to capital allocation, and the revenue expected from ORLADEYO, the company expects to achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon. Conference Call and Webcast BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; risks related to the reduction in size of BioCryst’s R&D organization; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com BIOCRYST PHARMACEUTICALS, INC.CONSOLIDATED FINANCIAL SUMMARY(In thousands, except per share) Statements of Operations (unaudited) Three Months EndedDecember 31, Twelve Months EndedDecember 31, 2023 2022 2023 2022 Revenues: ORLADEYO$90,883 $70,735 $325,990 $251,633 Other 2,518 8,810 5,422 19,194 Total revenues 93,401 79,545 331,412 270,827 Expenses: Cost of product sales 1,557 2,383 4,481 6,408 Research and development 70,052 73,207 216,566 253,297 Selling, general and administrative 64,382 50,153 213,894 159,371 Royalty 80 113 180 186 Total operating expenses 136,071 125,856 435,121 419,262 Loss from operations (42,670) (46,311) (103,709) (148,435) Interest and other income 4,465 2,704 15,777 5,127 Interest expense (24,583) (26,458) (108,239) (99,092)Loss on extinguishment of debt — — (29,019) — Foreign currency losses, net (374) (1,400) (1,039) (1,983)Loss before income taxes$(63,162) $(71,465) $(226,229) $(244,383)Income tax (benefit) expense (1,431) 76 310 2,733 Net loss$(61,731) $(71,541) $(226,539) $(247,116) Basic and diluted net loss per common share$(0.31) $(0.38) $(1.18) $(1.33) Weighted average shares outstanding 201,409 186,922 192,198 185,908 Balance Sheet Data (in thousands) December 31, 2023(unaudited) December 31, 2022(Note 1)Cash, cash equivalents and investments$388,987 $442,387 Restricted cash 1,804 1,472 Receivables 56,950 50,599 Total assets 516,960 550,000 Secured term loan 303,231 231,624 Royalty financing obligation 531,599 501,655 Accumulated deficit (1,681,159) (1,454,620)Stockholders’ deficit (455,528) (294,597)Shares of common stock outstanding 205,771 187,906 Note 1: Derived from audited financial statements Reconciliation of Adjusted Net Income and Adjusted Diluted Earnings Per Share (in thousands) Three Months EndedDecember 31, Twelve Months EndedDecember 31, 2023 2022 2023 2022 GAAP net loss$(61,731) $(71,541) $(226,539) $(247,116)Less: One-time loss on extinguishment of Athyrium term loans — — (29,019) — Less: One-time R&D restructuring expense (3,380) — (3,380) — Less: One-time cost associated with expensing previously capitalized costs due to postponement of Discovery Center (AL) expansion (1,988) — (1,988) — Adjusted net loss$(56,363) $(71,541) $(192,152) $(247,116) GAAP basic and diluted net loss per common share$(0.31) $(0.38) $(1.18) $(1.33) Adjusted basic and diluted net loss per common share$(0.28) $(0.38) $(1.00) $(1.33)

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Indication	Highest Phase	Country/Location	Organization	Date
C3 glomerulopathy	Phase 2	FR	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
C3 glomerulopathy	Phase 2	IT	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
C3 glomerulopathy	Phase 2	ES	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
C3 glomerulopathy	Phase 2	GB	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
glomerulonephritis chronic complement component 3 glomerulopathy	Phase 2	FR	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
glomerulonephritis chronic complement component 3 glomerulopathy	Phase 2	IT	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
glomerulonephritis chronic complement component 3 glomerulopathy	Phase 2	ES	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
glomerulonephritis chronic complement component 3 glomerulopathy	Phase 2	GB	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
Glomerulonephritis, IGA	Phase 2	FR	BioCryst Pharmaceuticals, Inc.	18 Feb 2022
Glomerulonephritis, IGA	Phase 2	IT	BioCryst Pharmaceuticals, Inc.	18 Feb 2022

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04702568 (CTgov)	Phase 2	Hemoglobinuria, Paroxysmal	19	BCX9930	qbytcvxpmm(ckamzuniju) = dsvgyexfpx zfutcsuqkl (agupmgwdfw, chwmqwlyio - odhzcifutx) View more	-	13 Dec 2024
NCT05162066 (RENEW, CTgov)	Phase 2	Idiopathic Membranous Glomerulonephritis \| C3 glomerulopathy \| Glomerulonephritis, IGA ... View more	2	BCX9930	pxrvfabthe(dbnnykbcgk) = hwtuzutict icofehjypb (nnsbjqoahw, uoncpbvcln - gxixqqtyeh) View more	-	02 May 2024
NCT04330534 \| NCT04702568 (BCX9930-101, EHA2022) Manual	Phase 1/2	Hemoglobinuria, Paroxysmal	16	BCX 9930	aaibfubegv(jgjakqiavo) = kritqdfzyn tbevssenye (zolcmuvjwv ) View more	Positive	12 May 2022
NCT04330534 (BCX9930-101, EHA2022)	Phase 1/2	Hemoglobinuria, Paroxysmal	16	BCX9930 monotherapy at 400 mg or 500 mg BID	kjkyocpnrd(ziozvibqrm) = ncgjnhmgpa puhpiwgjis (hmdlfcqvnx, 3.8%) View more	-	12 May 2022
NCT04330534 (BCX9930-101, ASN2021)	Phase 1/2	Disorder of Complement	-	BCX9930 50mg Q12h	muwpgkrebc(mmgsflffgv) = jyngqmfghz aaefxltrbg (vhqotdrogx )	Positive	27 Oct 2021
NCT04330534 (BCX9930-101, ASN2021)	Phase 1/2	Disorder of Complement	-	BCX9930 100mg Q12h	muwpgkrebc(mmgsflffgv) = mwranbkwmb aaefxltrbg (vhqotdrogx )	Positive	27 Oct 2021
NCT04330534 (BCX9930-101, EHA2021)	Phase 1/2	Hemoglobinuria, Paroxysmal	10	BCX9930	ybxpjxjzma(sdbjddmqld) = qdxlpqbfds zplszvbxkg (amgyfaytbu ) View more	Positive	09 Jun 2021
NCT04330534 (BCX9930-101, EHA2021)	Phase 1/2	Hemoglobinuria, Paroxysmal	6	BCX9930	ckvzuyetzn(gipqizrlep) = qpiiosefgo ppfyiepgof (iixlqbtxua ) View more	-	09 Jun 2021
NCT04330534 (ASH 12020 \| ASH 22020) Manual	Phase 1	Hemoglobinuria, Paroxysmal	108	BCX-9930	ilrbsypdul(lwanxrlvwe) = None udcpwisalr (mvdyhsteox ) View more	Positive	05 Nov 2020

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